Marc Boutin, Novartis’ global head of patient engagement, outlines the company’s “bold vision” of patient engagement and the actions that need to be taken for CAR-T patients in the immediate, short- medium-, and long-term. This piece forms part of our special section, InFocus: Patient Engagaement in the Era of CAR-T.


“How are you today?” For patients, this question may be a challenge. Honest answers can stop a conversation straight away. I know what I am talking about. 25 years ago, a series of tragic diagnoses hit my family.

Over a short period, all diagnosed loved ones passed from cancer, HIV and other diseases. A decade later I became a patient myself, suffering from cancer and an autoimmune disease. I could feel the pain of giving a polite answer to the conversational “how are you today?” question myself.

Conversational questions have a role in daily life. But they become obsolete in building deeper relationships. When I started my career as a patient advocate, I often felt that regulators and sponsors reached out to me with such conversational questions, usually shortly before – or around – a drug’s approval. I had the impression that they were rather seeking confirmation for their assumptions than real input.

It took a lot of effort, but gradually, groups like the National Health Council, succeeded in changing the ecosystem. Today, the FDA recognizes that patients are as much experts in their diseases as researchers and physicians. When I joined Novartis in 2020, my commitment was to bring the spirit of this patient-centric movement to the private sector.

Together with the leadership of Novartis and my team, we have created a bold vision towards patient engagement.

Today, the FDA recognizes that patients are as much experts in their diseases as researchers and physicians

Our vision puts a holistic and consistent engagement of patients across the whole lifecycle of medicines at the center of who we are and what we do. We will co-create patient relevant endpoints and co-design our clinical trial protocols with the community. And we aim to generate meaningful patient insights prior to defining our brand strategies.

This vision is of special value when we look at CAR-T therapies. In this area, patients are truly central to all activities as a patient’s own cells are the starting material for every manufacturing process and – after genetic modification – the essence of the final product.

CAR-T therapies are uniquely complex given their mode of action, their circular manufacturing model and the treatment modality as one-time therapies with a long-time therapeutic effect that is even considered potentially curative by some. Each of these aspects has a profound impact on how we engage with patients and while some require immediate action, others demand endurance and long-term engagement.


CAR-T – the need for immediate, short- medium- and long-term engagement with the community

Immediate: Counter the pandemic impact

The most immediate need for patients around the globe is to counter the detrimental effects of the pandemic. COVID19 prevented many patients from seeking prevention and therapy. In the CAR-T space, there was a drop in referrals of CAR-T eligible patients to the specialized CAR-T treatment centers. This is worrying, as most CAR-T eligible patients have no other viable treatment option. We even heard stories of patients who decided against CAR-T treatment to avoid blocking ICU beds that could potentially be used for COVID patients. However, most CAR-T patients will never need an ICU bed as treatment of side-effects (such as the infamous cytokine-release-syndrome) has improved enormously since the early days. As the world returns to a “new normal”, it will be paramount to ensure that the level of cancer prevention and appropriate treatment also returns to “normal. For many patient advocacy groups, the pandemic also had severe consequences, including a loss of fundraising opportunities that are often vital. Given the fundamental role these groups play in the healthcare ecosystem, these challenges need to be addressed jointly by the public and private sector.


Short term: Build awareness and understanding of rapidly changing treatment paradigms

In the short term, building awareness and understanding of the rapidly changing treatment paradigms in blood cancers is a priority. CAR-T therapies are a very powerful tool in treating advanced, aggressive blood cancers. For the individual patient, their full therapeutic benefit depends on many factors that need to be understood and put in context with other treatment options. One example is the overall fitness of a patient’s T-cells – a key success factor in CAR-T therapies. As the science and medicine is complex, patient advocacy groups play a central role in this area. For Novartis and other sponsors, this space is a sensitive area and requires utmost responsibility. Our country teams received requests from patients and caregivers who asked for access to CAR-T therapies, in approved as well as in non-approved indications. Often, such requests were triggered by highly visible TV or newspaper reports. We need to strike a balance between providing accurate information on a transformative therapy and the responsibility to manage expectations for patients whose cancer cannot be treated with CAR-T therapies. The role of the patient engagement function in Novartis is to be a critical voice in all our internal discussions and decision-making processes, ensuring that this balance is well kept.


MediumtermExpand access and build the real-world dataset, complemented with meaningful PROs

As the sector continues to commercialize CAR-T therapies around the globe and in earlier lines of treatment, there is an ever growing demand from patients, healthcare providers and payers to understand the true long-term benefit of the therapy through real-world evidence. For one-time therapies such as CAR-T and gene-therapies, this space holds some new challenges for us as well as for scientists and patient advocacy groups. How can patients, who may feel “cured” after a successful CAR-T therapy be encouraged to share their long-term health data for such longitudinal studies? How will the situation evolve for young patients, who may be treated while being a teenager before leaving their homes to work or study abroad? These questions can only be tackled in true collaboration with the patient community and again, patient advocacy groups do play a vital role.

Additionally, there is a need to build a set of meaningful patient-reported outcomes (PROs) which complement the “hard”, medical data. As CAR-T therapies may increasingly be used in earlier lines of therapy, the quality of life before, during and after therapy will be an important parameter. Complete remission (CR), progression-free survival (PFS) and event-free-survival (EFS) are key – but for patients it will be paramount, “how” these outcomes are achieved. Compared to some standard-of-care interventions (e.g. stem cell transplants), CAR-T therapies do not require long hospitalization periods. We all assume that this is of real patient benefit (especially for children and young adults who may miss on education during treatment) but there is a lack of validated evidence. Organizations such as the Global Lymphoma Coalition are already deeply involved in this area. Contributing to building this evidence will be a key objective of our patient engagement teams in Novartis and we are supporting the collection of such insights, for example through educational grants given to patient advocacy groups.


Long-termCo-create research and clinical trials for CAR-T therapies

As the field matures, we need to ensure that clinical trial set-ups and protocols are co-created with the community. In Novartis, our objective is to do this for all newly starting trials and potentially even earlier, as we define our research and development strategy. This is the core of our big picture vision for patient engagement – to be the first pharmaceutical company to consistently and systematically engage patients across the medicine’s lifecycle.

I firmly believe that the potential of CAR-T therapies will continue to grow in indications outside of hematology, potentially even outside of oncology. This will require a constant, open, transparent, and responsible collaboration with patient communities. Only together can we ensure that research programs target the right indications with appropriate trials and harvest the full benefit of patient insights.

As the pandemic will hopefully come to an end soon, I truly look forward to meeting many representatives of the patient community in real life again. I hope, they will give me honest answers to the non-conversational question “What’s your opinion on Novartis’ collaboration with the patient community?”